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Characterization of the Clinical-epidemiological Profile of Patients With SMA5q Types II and III: Observational Study

NCT04404764 · Status: COMPLETED · Type: OBSERVATIONAL · Enrollment: 155

Last updated 2021-04-27

No results posted yet for this study

Summary

This study aims to characterize the clinical-epidemiological profile and baseline characteristics of patients with spinal muscular atrophy (SMA) 5q types II and III in follow-up at the Brazilian Unified Public Health System (SUS). The study data will be based on patients´ medical records from several Brazilian public hospitals, which will be defined by the Brazilian Ministry of Health (MS).

Conditions

Interventions

DRUG

Nusinersen Injectable Product

The patient´s treatment is provided by SUS. The following dosage scheme of nurinersen is the one approved by ANVISA (Brazilian National Health Surveillance Agency): Nusinersen, 12 mg (5 mL) on days 0 (zero), 14 and 28. A fourth dose will be on day 63 with a maintenance dose once every 4 (four) months. The study will NOT have direct influence on the care received by patients. Data on adherence, interventions, hospitalizations, mechanical ventilation, procedures and adverse events will be obtained from the patients´ medical records.

Sponsors & Collaborators

  • Ministry of Health, Brazil

    collaborator OTHER_GOV

  • Hospital Israelita Albert Einstein

    lead OTHER

Principal Investigators

  • Otávio Berwanger, PhD · Hospital Israelita Albert Einstein

  • Vanessa Teich, PhD · Hospital Israelita Albert Einstein

  • Edmar Zanoteli, PhD · University of Sao Paulo

  • Elice Batista, PhD · Hospital Israelita Albert Einstein

Eligibility

Min Age
6 Months
Sex
ALL
Healthy Volunteers
Yes

Timeline & Regulatory

Start
2020-05-27
Primary Completion
2021-04-23
Completion
2021-04-23

Countries

  • Brazil

Study Locations

More Related Trials

Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04404764 on ClinicalTrials.gov